Prescription medicine
Miglustat Brand names: Opfolda, Zavesca, Yargesa
Miglustat (Zavesca) is a prescription medicine with an FDA approval on record since 2003. FDA's NDC Directory lists it from 4 generic labelers.
- Generic listed
Check it at the source: the miglustat FDA label on DailyMed (effective April 20, 2023, Actelion Pharmaceuticals US, Inc.); the NDA 21348 record on Drugs@FDA; every miglustat label on DailyMed.
Highlights of the record
FDA label of April 20, 2023; NDC Directory, Drugs@FDA and Orange Book, October 8, 2026
Used for
From the label: indications and usageZAVESCA is a glucosylceramide synthase inhibitor indicated as monotherapy for treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option ( 1.1 ).
Forms and strengths
- 65 mg
- 100 mg
Capsule.
On the market
- Earliest approval on file
- July 31, 2003 Zavesca, Actelion; Drugs@FDA’s file, where older or withdrawn approvals can be missing
- Generic approvals
- 4 first April 17, 2018; 3 with a marketed status in Drugs@FDA
- Labelers listed (NDC)
- 7 of which 4 under generic applications, 2 under brand applications, repackagers included
- Last listed patent
- September 16, 2038 Opfolda capsules; not a generic launch date
Supply and safety
- Pharmacy cost (NADAC)
- Not in the survey
- Shortage (FDA)
- No current entry
- Recalls by its makers (FDA)
- No recall recorded
- FAERS reports
- 1,154 through July 30, 2026
What is miglustat used for?
From the label: indications and usageZAVESCA is a glucosylceramide synthase inhibitor indicated as monotherapy for treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option ( 1.1 ). 1.1 Type 1 Gaucher Disease ZAVESCA is indicated as monotherapy for the treatment of adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option (e.g. due to allergy, hypersensitivity, or poor venous access).
FDA pharmacologic class: Enzyme Stabilizer and Glucosylceramide Synthase Inhibitor.
Source: FDA drug label (Zavesca), Actelion Pharmaceuticals US, Inc., effective April 20, 2023 (SPL set 817892d1-ee12-4632-85fc-57ccdf16d7b8), via openFDA; the full label on DailyMed
Is there a generic for miglustat (Zavesca)?
The FDA approved the first generic miglustat on April 17, 2018. 4 generic applications are approved; 3 of them carry a current (not discontinued) marketing status in Drugs@FDA; that status is not evidence that the generic is on sale.
FDA's NDC Directory lists miglustat from 4 labelers under a generic (ANDA) application and from 2 brand labelers. A listing means the company has registered the product for sale; it does not show that every pharmacy stocks it.
The Orange Book lists no patent for Zavesca capsules; a patent listed only for Opfolda capsules runs to September 16, 2038. A patent or exclusivity date is not the date a generic goes on sale: court cases and settlements move it either way.
- 2003 earliest approval on file
- 2018 first generic approved
- 2038 last listed patent
Source: Drugs@FDA, FDA NDC Directory and the Orange Book (openFDA bulk files, October 8, 2026)
What does the miglustat label say about other medicines?
The miglustat label names 3 other medicines in its interaction, contraindication or warning sections. The labels of 1 other medicine name miglustat. The sentences are the labels' own; a label lists what its studies and reports found, not every interaction.
| Medicine named | Section | What the label says |
|---|---|---|
| Cipaglucosidase alfa | contraindications | CONTRAINDICATIONS OPFOLDA in combination with Pombiliti is contraindicated in Pregnancy [see Use in Specific Populations (8.1 )] Pregnancy. |
| Imiglucerase | drug interactions | DRUG INTERACTIONS While co-administration of ZAVESCA appeared to increase the clearance of imiglucerase by 70%, these results are not conclusive because of the small number of patients studied and because patients took variable doses of imiglucerase [see Clinical Pharmacology (12.3) ]. |
| Loperamide | warnings and precautions | Diarrhea decreased over time with continued ZAVESCA treatment, and may respond to individualized diet modification (e.g., reduction of sucrose, lactose and other carbohydrate intake), to taking ZAVESCA between meals, and/or to anti-diarrheal medications, most commonly loperamide. |
Source: FDA drug label (Zavesca), Actelion Pharmaceuticals US, Inc., effective April 20, 2023 (SPL set 817892d1-ee12-4632-85fc-57ccdf16d7b8), via openFDA; the full label on DailyMed
What do FAERS reports show for miglustat?
The FDA Adverse Event Reporting System holds 1,154 reports that list miglustat among the medicines taken, through July 30, 2026, each a report of something that happened, not proof the medicine caused it; 319 are coded as a death and 418 as a hospital stay. Counts follow how widely a medicine is used and watched, and they are not a measure of how risky it is.
Reports received each year
The most reported reactions
Source: FDA Adverse Event Reporting System (FAERS) via openFDA, reports through July 30, 2026
What next?
- Other glucosylceramide synthase inhibitors
- the pharmacologic class, with prices and generics
- Miglustat side effects
- the label's list beside FAERS reports
- Check an interaction
- what two or more labels say about each other